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Chance or necessity?

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Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. / Baum, Christopher; von Kalle, Christof; Staal, Frank J T; Li, Zhixiong; Fehse, Boris; Schmidt, Manfred; Weerkamp, Floor; Karlsson, Stefan; Wagemaker, Gerard; Williams, David A.

in: MOL THER, Jahrgang 9, Nr. 1, 01.2004, S. 5-13.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Review › Forschung

Harvard

Baum, C, von Kalle, C, Staal, FJT, Li, Z, Fehse, B, Schmidt, M, Weerkamp, F, Karlsson, S, Wagemaker, G & Williams, DA 2004, 'Chance or necessity? Insertional mutagenesis in gene therapy and its consequences', MOL THER, Jg. 9, Nr. 1, S. 5-13. https://doi.org/10.1016/j.ymthe.2003.10.013

APA

Baum, C., von Kalle, C., Staal, F. J. T., Li, Z., Fehse, B., Schmidt, M., Weerkamp, F., Karlsson, S., Wagemaker, G., & Williams, D. A. (2004). Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. MOL THER, 9(1), 5-13. https://doi.org/10.1016/j.ymthe.2003.10.013

Vancouver

Baum C, von Kalle C, Staal FJT, Li Z, Fehse B, Schmidt M et al. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. MOL THER. 2004 Jan;9(1):5-13. https://doi.org/10.1016/j.ymthe.2003.10.013

Bibtex

@article{2a4d0b5c2e604b7e93554b334560ce4a,
title = "Chance or necessity?: Insertional mutagenesis in gene therapy and its consequences",
abstract = "Recently, unusual forms of leukemias have developed as complications following retroviral transfer of potentially therapeutic genes into hematopoietic cells. A crucial component in the pathogenesis of these complications was the upregulation of a cellular proto-oncogene by random insertion of the retroviral gene transfer vector. These findings have great implications for the genetic manipulation of somatic stem cells in medicine. This review discusses the extent to which the random oncogene activation may have required disease-specific stimuli of the transgene and the hematopoietic milieu to become leukemogenic. Based on these considerations, we propose approaches to risk prediction and prevention.",
keywords = "Adaptor Proteins, Signal Transducing, Animals, DNA-Binding Proteins/biosynthesis, Genetic Therapy/adverse effects, Genetic Vectors, Hematopoietic Stem Cell Transplantation, Humans, LIM Domain Proteins, Leukemia, T-Cell/etiology, Leukemia-Lymphoma, Adult T-Cell/etiology, Metalloproteins/biosynthesis, Mice, Mutagenesis, Insertional, Proto-Oncogene Mas, Proto-Oncogene Proteins, Proto-Oncogenes/genetics, Retroviridae/genetics, Risk Assessment, Up-Regulation/genetics",
author = "Christopher Baum and {von Kalle}, Christof and Staal, {Frank J T} and Zhixiong Li and Boris Fehse and Manfred Schmidt and Floor Weerkamp and Stefan Karlsson and Gerard Wagemaker and Williams, {David A}",
year = "2004",
month = jan,
doi = "10.1016/j.ymthe.2003.10.013",
language = "English",
volume = "9",
pages = "5--13",
journal = "MOL THER",
issn = "1525-0016",
publisher = "NATURE PUBLISHING GROUP",
number = "1",

}

RIS

TY - JOUR

T1 - Chance or necessity?

T2 - Insertional mutagenesis in gene therapy and its consequences

AU - Baum, Christopher

AU - von Kalle, Christof

AU - Staal, Frank J T

AU - Li, Zhixiong

AU - Fehse, Boris

AU - Schmidt, Manfred

AU - Weerkamp, Floor

AU - Karlsson, Stefan

AU - Wagemaker, Gerard

AU - Williams, David A

PY - 2004/1

Y1 - 2004/1

N2 - Recently, unusual forms of leukemias have developed as complications following retroviral transfer of potentially therapeutic genes into hematopoietic cells. A crucial component in the pathogenesis of these complications was the upregulation of a cellular proto-oncogene by random insertion of the retroviral gene transfer vector. These findings have great implications for the genetic manipulation of somatic stem cells in medicine. This review discusses the extent to which the random oncogene activation may have required disease-specific stimuli of the transgene and the hematopoietic milieu to become leukemogenic. Based on these considerations, we propose approaches to risk prediction and prevention.

AB - Recently, unusual forms of leukemias have developed as complications following retroviral transfer of potentially therapeutic genes into hematopoietic cells. A crucial component in the pathogenesis of these complications was the upregulation of a cellular proto-oncogene by random insertion of the retroviral gene transfer vector. These findings have great implications for the genetic manipulation of somatic stem cells in medicine. This review discusses the extent to which the random oncogene activation may have required disease-specific stimuli of the transgene and the hematopoietic milieu to become leukemogenic. Based on these considerations, we propose approaches to risk prediction and prevention.

KW - Adaptor Proteins, Signal Transducing

KW - Animals

KW - DNA-Binding Proteins/biosynthesis

KW - Genetic Therapy/adverse effects

KW - Genetic Vectors

KW - Hematopoietic Stem Cell Transplantation

KW - Humans

KW - LIM Domain Proteins

KW - Leukemia, T-Cell/etiology

KW - Leukemia-Lymphoma, Adult T-Cell/etiology

KW - Metalloproteins/biosynthesis

KW - Mice

KW - Mutagenesis, Insertional

KW - Proto-Oncogene Mas

KW - Proto-Oncogene Proteins

KW - Proto-Oncogenes/genetics

KW - Retroviridae/genetics

KW - Risk Assessment

KW - Up-Regulation/genetics

U2 - 10.1016/j.ymthe.2003.10.013

DO - 10.1016/j.ymthe.2003.10.013

M3 - SCORING: Review article

C2 - 14741772

VL - 9

SP - 5

EP - 13

JO - MOL THER

JF - MOL THER

SN - 1525-0016

IS - 1

ER -